Kite Pharma: Focused on the Cure. Arie Belldegrun, MD, FACS Chairman, President, & Chief Executive Officer

Transcription

1 Kite Pharma: Focused on the Cure Arie Belldegrun, MD, FACS Chairman, President, & Chief Executive Officer

2 Forward Looking Statements/Safe Harbor To the extent statements contained in this presentation are not descriptions of historical facts regarding Kite Pharma, Inc. ( Kite, we, us, or our ), they are forward-looking statements reflecting management s current beliefs and expectations. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry s actual results, levels or activity, performance, or achievements to be materially different from those anticipated by such statements. You can identify forward-looking statements by words such as anticipate, believe, could, estimate, expect, intend, may, plan, potential, predict, project, should, will, would or the negative of those terms, and similar expressions that convey uncertainty of future events or outcomes. Forward-looking statements contained in this presentation include, but are not limited to, statements regarding: (i) the success and timing of our product development activities and clinical trials; (ii) the ability and willingness of the National Cancer Institute (NCI) to continue research and development activities relating to our product candidates; (iii) our ability to obtain and maintain regulatory approval of KTE-C19 and any other product candidates; (iv) our ability to further develop and commercialize our product candidates; (v) our plans to research, discover and develop additional product candidates, including through our subsidiary Kite Pharma EU, and next generation product candidates, including a next-generation CAR with an on/off switch; (vi) our and our partners ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process; (vii) the size and growth potential of the markets for our product candidates, and our ability to serve those markets; (viii) the rate and degree of market acceptance of our product candidates; (ix) our ability to attract and retain key scientific or management personnel; (x) the anticipated timing of clinical data availability; (xi) the anticipated timing of commercial launch of KTE-C19; (xii) our plans to expand geographically; (xiii) our ability to meet the milestones set forth herein and (xiv) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates. Various factors may cause differences between Kite's expectations and actual results as discussed in greater detail in Kite's filings with the Securities and Exchange Commission (SEC), including without limitation in its Quarterly Report on Form 10- Q filed with the SEC on May 9, Except as required by law, we undertake no obligation to publicly update any forwardlooking statements, whether as a result of new information, future events or otherwise. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy securities, nor shall there be any sale of securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. 2

3 Kite: Focused on the Cure 3 Changing Empowering Utilizing Advancing Optimizing Building the paradigm of cancer treatment a patient s own immune system to combat cancer two technology platforms to address both hematologic and solid tumors our lead product candidate in four pivotal studies and preparing for 2017 commercial launch and automating in-house manufacturing to bring cell therapies to the broad market a pipeline of future cancer therapies with innovative internal R&D and collaborations with select industry leaders

4 Developing The Ultimate Personalized Cancer Immunotherapy ENGINEERED AUTOLOGOUS CELL THERAPY (eact ) Apheresis Manufacturing Process Infusion 4

5 Dual Platform Targets Both Hematological and Solid Cancers Chimeric Antigen Receptor (CAR) Targets molecules on the cell surface T Cell Receptor (TCR) Targets molecules at or below the cell surface 5

6 Kite Pharma: An Industry Leading Pipeline of CAR and TCR Product Candidates CHIMERIC ANTIGEN RECEPTOR (CAR) PRE-IND PHASE 1 PHASE 2 KTE-C19 DLBCL, PMBCL, TFL (ZUMA-1) T CELL RECEPTOR (TCR) PRE-IND PHASE 1 PHASE 2 MAGE A3/A6 Solid tumors KTE-C19 MCL (ZUMA-2) MAGE A3 Solid tumors KTE-C19 Adult & Pediatric ALL (ZUMA-3, ZUMA-4) KTE-C19 2 nd Wave of ZUMA Studies Combination, FL, CLL Human Anti-CD19 Heme malignancies Amgen Multi-Target Collaboration Heme malignancies Amgen Multi-Target Collaboration Solid tumors HPV-16 E6 Cervical and head & neck cancer HPV-16 E7 Cervical and head & neck cancer NY-ESO-1 Solid tumors SSX2 Solid tumors KRAS KRAS mutation tumors NEO-ANTIGENS Solid tumors 6 DLBCL = diffuse large B cell lymphoma PMBCL = primary mediastinal B cell lymphoma TFL = transformed follicular lymphoma MCL = mantle cell lymphoma ALL = acute lymphoblastic leukemia FL = follicular lymphoma CLL = chronic lymphocytic leukemia

7 Kite: Investigating Targets with Broad Clinical Applicability CD19 Non-Hodgkin Lymphoma Leukemias Kite Targets from Amgen Collaboration AML MM Kidney Lung CARs TCRs MAGE A3/A6 NY-ESO-1 HPV-16 E6/E7 SSX2 KRAS Neo-Antigens Lung Pancreatic Gastric Cervical Head & Neck Anal Cancer Urothelial Sarcoma Lung HCC Melanoma Prostate Pancreatic CRC Lung Potentially all types of cancer Breast Melanoma Sarcoma 7

8 Executive Management and Scientific Team Have a Proven Track Record of Success Arie Belldegrun, MD, FACS Chairman, President and CEO Cynthia M. Butitta Chief Operating Officer Paul Jenkinson Chief Financial Officer David D. Chang, MD, PhD EVP R&D, Chief Medical Officer Shawn C. Tomasello Chief Commercial Officer Tim Moore EVP, Technical Operations Helen S. Kim EVP, Business Development Margo R. Roberts, PhD Chief Scientific Officer Jeff Wiezorek, MD, MS SVP, Clinical Development Executive Management Chiron Owen N. Witte, MD (Chairman) UCLA Scientific Advisory Board James Allison, PhD The University of Texas MD Anderson Cancer Center James Economou, MD, PhD UCLA Zelig Eshhar, PhD Tel Aviv Sourasky Medical Center & Weizmann Institute of Science Donald Kohn, MD UCLA Ton N. M. Schumacher, PhD Chief Scientific Officer Ronald Levy, MD Stanford School of Medicine Antoni Ribas, MD, PhD UCLA Padmanee Sharma, MD, PhD The University of Texas MD Anderson Cancer Center Inder Verma, PhD The Salk Institute Special Advisor Steven A. Rosenberg, MD, PhD NCI/KITE CRADA Kite Pharma EU 8

9 Leveraging Academic and Industry Collaborations for Next Generation Technologies Strategic Collaborations Surgery Branch Experimental Transplantation & Immunology Branch TCR Enabling Technologies CAR 9

10 Significant Accomplishments Initiated four KTE-C19 pivotal studies in DLBCL, MCL, adult, and pediatric ALL under two separate company INDs Received Breakthrough Therapy Designation for KTE-C19 under company IND in the US and PRIME Designation in the EU Secured Orphan Drug Designation for KTE-C19 in the US and EU for all our hematological indications Established European operations with T-Cell Factory acquisition, NKI agreement and launched Kite EU Built out the company with the addition of over 100 new hires, including the Chief Commercial Officer and EVP of Technical Operations 10

11 11 DELIVER IN HEMATOLOGY Four pivotal studies for KTE-C19 Second wave of KTE- C19 studies Commercial launch of KTE-C19 in 2017 planned FOCUS ON SOLID TUMORS 2016 IND for MAGE A3 CAR product candidates from Amgen collaboration TCR GENErator TM technology for additional TCR product candidates DEVELOP NEXT GEN. CELL THERAPY Combination therapy (Genentech) Modulating immune synapse using the vigd technology (Alpine IS) Fully automated process (GE Global Research) Building the Future of Cancer Therapy

12 Introducing Control to CAR through Synthetic Biology A SPLIT CAR design that allows for a DIALABLE, INSTANT, and, REVERSIBLE control of CAR activity, in the presence of a small molecule scfv, single-chain variable fragment; TCR, T cell receptor; ITAM, immunoreceptor tyrosine-based activation motif 32

13 Ongoing ZUMA Studies to Support Registration of KTE-C19 Study Phase Indication Status ZUMA-1 ZUMA-2 ZUMA-3 ZUMA-4 Phase 2 Pivotal (N=112) Phase 2 Pivotal (N=70) Phase 1/2 Pivotal (N=75) Phase 1/2 Pivotal (N=75) DLBCL PMBCL TFL MCL Adult ALL Pediatric ALL Additional studies in FL, CLL, earlier lines of DLBCL, and combination studies starting in 2016 Enrolling Interim Data * 2016 Primary Data 2017 Enrolling Data 2017 Phase 1/2 Enrolling P2 Data 2017 Phase 1/2 Enrolling P2 Data *If supported by data, plan to file Biologics License Application based on interim data

14 KTE-C19 Will Address Largest Unmet Need in NHL Incidence per year US new cases per year US deaths per year 26,000 15,700 10,000 4,600 6,000 1,400 DLBCL CLL ALL 14 *Ex-US incidence of DLBCL is 24,000 cases per year (France, Germany, Italy, Spain, UK, and Japan)

15 ZUMA-1 Phase 1 Clinical Data Show Ongoing CRs at 9-Month Follow-Up Efficacy 71% Objective Response Rate (n=5/7) 57% Complete Remissions (n=4/7) 3 Complete Remissions ongoing at 9 months Safety 1 patient with DLT (grade 4 CRS and neurotoxicity) died on study (unrelated to KTE-C19) 14% grade 3+ CRS 57% grade 3+ neurotoxicity With the exception of the patient with DLT, all grade 3 KTE-C19-related toxicities resolved Baseline Complete Response at Day 30 Neelapu et al, ASCO Annual Meeting

16 SCHOLAR-1: the first and largest patient-level meta-analysis of chemorefractory DLBCL SCHOLAR-1 is a retrospective analysis of 635 patients with chemorefractory DLBCL from 4 studies/institutions Patients with chemorefractory DLBCL have consistently poor outcomes regardless of refractory subgroup, line of therapy, and disease stage These data provide a historical benchmark for future studies in chemorefractory DLBCL Overall response rate 26% (18% PR & 8% CR) Median OS 6.6 months 17

17 NCI Data Demonstrating Breakthrough Efficacy in Aggressive NHL Group (n) Best Response ORR CR All Patients (41)* 78% 54% All Aggressive NHL (27) 70% 48% Aggressive NHL with low dose conditioning regimen (19) ** 68% 47% Aggressive NHL with low dose conditioning regimen and Kite manufacturing process (13) 69% 54% Generally reversible CAR-related grade 3-4 AEs CRS: All patients 30%, Kite manufacturing 31% Neurotoxicity: All patients 44%, Kite manufacturing 69% **All CRs (9) are still in CR *8 patients converted from PR to CR Data as of 11/30/15 Data from ASCO

18 DLBCL Patient 38 With an Ongoing CR That Had Progressed on R-ICE, GDP, and R-EPOCH Before treatment 10 months after treatment Before treatment 10 months after treatment 18 18

19 Manufacturing On Track for KTE-C19 Launch in 2017 In-house clinical manufacturing in full operation Commercial facility in close proximity to LAX airport Capacity to produce 4,000-5,000 patient therapies per year Modular design is scalable and cost effective Site to produce KTE-C19 and all TCR products 19

20 9 4 Ongoing Registration Studies in >40 Sites

21 TCRs Have the Potential to Access a Broad Spectrum of Therapeutic Tumor Targets Potential CAR Targets (~27% Human Proteome) Potential TCR Targets (All Human Proteome) 21

22 Proof of Concept for MAGE A3 TCR and HPV TCR PR: 85% Tumor Reduction Cervical Cancer Patient 100% Tumor Disappearance Pre-Treatment MAGE A3 is expressed in NSCLC, bladder, myeloma, SCC, melanoma, among others 3 responders (cervical, esophageal, and urothelial cancer) among 14 evaluable patients No off-target toxicities 12 Months HLA-DPB1*0401 present in 40% to 70% of Caucasian population Pre-Treatment 15 Months HPV infection is associated with 5% of all cancers globally Durable CR (22+ months) in cervical cancer patients treated with HPV reactive T cell therapy 1 HPV-16 E6 TCR Phase 1 study at NCI showed a significant response (-90%) in a patient with anal cancer HLA-A*0201 is present in 40% of the total US population 22 Lu, YCW, AACR Annual Meeting Stevanovic, 2015, J Clin Onc epub

23 Innovating the Next Generation of Products Target Selection Validate Target Selectivity Advance Cancer-Specific Targets CAR/TCR Optimization Improve Target Fidelity of CAR/TCR Constructs Modulate CAR/TCR Function in vivo Reduce Immunogenicity Manufacturing Automate Manufacturing Selectively Expand Potent T Cell Subsets ex vivo Tumor Microenvironment Overcome Immune Suppression Combination Therapy Introduce Gene Editing or Gene Modulation Clinical Development Optimize in vivo T Cell Expansion Improve Safety Management Identify Predictive Biomarkers for Safety and Efficacy 23

24 2016 Projected Milestones Report interim data for Phase 2 ZUMA-1 File KTE-C19 BLA on interim data Complete qualification/validation of manufacturing facility Initiate KTE-C19 clinical studies in Europe Initiate Phase 1b/2 combination study of Kite s KTE-C19 and Genentech s atezolizumab File IND for first Kite TCR product: MAGE A3 25

25 Kite: Focused on the Cure 25 Changing Empowering Utilizing Advancing Optimizing Building the paradigm of cancer treatment a patient s own immune system to combat cancer two technology platforms to address both hematologic and solid tumors our lead product candidate in four pivotal studies and preparing for 2017 commercial launch and automating in-house manufacturing to bring cell therapies to the broad market a pipeline of future cancer therapies with innovative internal R&D and collaborations with select industry leaders